FINAL PROGRAM - American Society of Gene & Cell Therapy

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13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 37Program ScheduleEducation Session 1305:30 pm - 7:00 pmRoom: Marriott Ballroom Salon 1Emerging Field Review: Progress Towards Using ES/iPS Cells for Regenerative MedicineThe excitement over embryonic stem cells and induced pluripotent stem cells is that they are a potential source of cells for tissues in which tissue specific stem cells haveeither not been identified, not easily isolated, or not easily expanded. That pluripotent stem cells can now be generated from somatic cells, without the destruction of anembryo, removes a major ethical barrier to their use. Nonetheless, a continued barrier to the translation of pluripotent stem cells to clinical use is to expand and differentiatethem in both a safe and quantitatively sufficient manner that they can be used for tissue specific regenerative medicine applications. This educational session willfocus on the progress that has been made in generating sufficient numbers of safe tissue specific cells towards this end.Wednesday, May 19 thChairMatthew H. Porteus, MD, PhDSpeakersRonald D.G. McKay, PhDElias T. Zambidis, MD, PhDGeneration of HSC and Red Blood Cells from Normal and Hemoglobinopathic iPSCEvan Y. Snyder, MD, PhD, FAAPCross-Talk and Developmental Programs in Stem Cell Biology – A Key to Regenerative MedicineEducation Session 1315:30 pm - 7:00 pmRoom: Maryland SuiteEmerging Field Review: Targeted IntegrationUncontrolled insertion of foreign DNA into the human genome may have deleterious consequences, and is a serious safety concern in the clinical application of genetherapy. New molecular tools are being developed to target transgene integration to specific sites in the genome and increase the frequency of homologous recombination,with the purpose of achieving gene correction or replacement in the original genomic environment. These advances include the use of Zn-finger nucleases and homingendonucleases, and the delivery of homologous recombination cassettes by viral and non-viral vectors. They will be discussed in the context of developing better and safergene transfer technology.ChairFulvio Mavilio, PhDSpeakersJ. Keith Joung, MD PhDEngineered Zinc Finger Nucleases for Targeted Genome ModificationEngineered zinc finger nucleases (ZFNs) form the basis of a broadly applicable approach for inducing targeted alterations with high efficiency in human and other mammaliancells. This session will provide an introduction to this important technology by: (1) discussing methods for engineering customized ZFNs including OligomerizedPool ENgineering (OPEN) and modular assembly; (2) describing how ZFNs have been used to introduce knockout mutations, insertions and other specific alterations vianon-homologous end-joining and homologous recombination; and (3) considering future experiments and goals for advancing the use of ZFNs for therapeutic and researchapplications.Andrew M. Scharenberg, MDHoming Endonucleases as Tools for Targeted IntegrationThe idiosyncratic nature of side effects resulting from random integration of promoter/gene cassettes in the context of viral vector-mediated gene therapy has led toincreasing interest in targeted integration as an alternative approach. A promising methodology for targeted integration involves application of rare cleaving nucleases toinduce site specific homologous recombination. Homing endonucleases are an important class of rare cutting nucleases whose potential applications in targeted integrationare only now emerging. Homing endonuclease biology, properties, engineering, and applications in targeted integration will be reviewed.
38American Society of Gene & Cell TherapyProgram ScheduleWednesday, May 19 thDavid W. Russell, MD, PhDAAV-Mediated Gene TargetingAAV vectors efficiently recombine with homologous chromosomal sequences in mammalian cells. Up to 1% of the entire cell population exposed to vector particlesundergoes gene targeting under optimal conditions. Insertions, deletions and substitutions can all be introduced at precise chromosomal locations with high fidelity. Thetechnique works well in many cell types, including transformed cell lines, stem cells, and even in vivo in murine hepatocytes. Importantly, the gene targeting occurswithout genotoxicity and endonucleases are not required to obtain high targeting frequencies.Education Session 1325:30 pm - 7:00 pmRoom: Thurgood Marshall WTopical Review: Gene Delivery to the HeartHeart disease is one of the leading causes of morbidity and mortality worldwide. While molecular causes are known for many heart diseases, it requires effective heartgene delivery methods to translate these findings into therapy. Because of the dynamic nature of the heart physiology and the unique anatomic location, cardiac genetransfer has been a great challenge. Significant progress has been made over the last few years. In this session, a historical overview of heart gene transfer will be provided.Considering the importance of the large animal model in translational medicine, special emphasis will be given to cardiac gene delivery in the canine and porcinemodels.ChairDongsheng Duan, PhDSpeakersDongsheng Duan, PhDOverview of Heart Gene Transfer in MiceEfficient cardiac gene transfer holds great promise in treating heart diseases, a major health threat worldwide. Over the last decades, tremendous progresses have beenmade in delivering the marker and/or therapeutic genes to the mouse heart. This presentation will provide a historic review of the literature and an update of the currentdevelopments.Roger J. Hajjar, MDGene Therapy for Heart Failure: New Targets and New VectorsDespite the proliferation of therapies, congestive heart failure remains a progressive disease where a desperate need for innovative rather than incremental therapies toreverse the course of ventricular dysfunction. Recent advances in understanding the molecular basis of myocardial dysfunction, together with the evolution of increasinglyefficient gene transfer technology; have placed heart failure within reach of gene-based therapies. One of the key abnormalities in both human and experimental HF isa defect in sarcoplasmic reticulum (SR) function, which is responsible for abnormal intracellular Ca2+ handling. Restoring SERCA2a levels has been shown to improvefunction, metabolism and/or survival in a number of experimental models of heart failure. Over the last ten years we have undertaken a program of targeting importantcalcium cycling proteins in experimental models of heart by somatic gene transfer. This has led to the completion of a first-in-man phase 1 clinical trial of gene therapy forheart failure using adeno-associated vector (AAV) type 1 carrying SERCA2a. In this Phase I trial, there was evidence of clinically meaningful improvements in functionalstatus and/or cardiac function which were observed in the majority of Neutralizing antibody negative patients at various time points. The safety profile of AAV genetherapy along with the positive biological signals obtained from this phase 1 trial has led to the recent completion of a phase 2 trial of AAV1.SERCA2a in NYHA class III/IV patients.Charles R. Bridges, MD, ScDMethods for Cardiac Gene DeliveryThis presentation will review the available methods of cardiac vector mediated gene delivery including those in use in current clinical trials. Methods considered will includeintracoronary injection, intramuscular injection, pressurized coronary venous retroinfusion, and cardiopulmonary bypass-mediated techniques. The relative strengths andweaknesses of these delivery methods will be discussed with reference to efficiency of transfection, cardiac versus collateral organ specificity and relative propensity toinduce a T-cell mediated immune response.EXHIBITOR PROSPECTUSfinal program
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